The mission of Columbus Children’s Foundation is driven by the urgency to accelerate equitable access to the most effective gene therapy solutions for children with ultra-rare genetic diseases.
Together we can change lives and ensure precious moments
are never lost.
Accelerating access to gene therapy for ultra-rare genetic disorders
The Columbus Children’s Foundation is driven by the urgency to advance gene therapy research to bring hope to children with ultra-rare genetic diseases.
Improving lives of children with ultra-rare diseases
Our goal is to see children going from wheelchairs to walking; from no speech to speaking, from lacking sight to seeing smiles; and from only receiving hugs to giving hugs.
Global Partner: Interventional NeuroTherapy Center, Brodno Hospital, Warsaw, Poland
Brodno Hospital provides avenues for advanced gene therapy treatments for the world’s rarest children. This is the first Interventional Neurology Center (INC) to open in Europe and the second in the world. Led by Krys Bankiewicz, doctors at the INC perform extremely precise operations to treat and cure children from around the world.
Marta M., Spain
Marta M. was just two months old when she was diagnosed as only the 100th child in Spain with Niemann Pick C disorder, an incurable neurodegenerative hereditary disease with a life expectancy of at best 20 years. Thanks to the support of the Fundacion Columbus, our European counterpart, Marta now has a life filled with hope.
The power of music
“We build bridges to help children born with ultra-rare diseases through the power of music. Just as a musical composition is a complex combination of perfectly tuned notes, so is the human genome. When in perfect harmony, both have amazing healing powers. Our worldwide concerts build awareness for children with ultra-rare diseases to raise needed funds.”
– Javier Garcia, CCF Board Trustee
Our latest news
Madrid, February, 2021.- A new treatment for the rare and fatal neurodegenerative disease Niemann Pick C is closer. University College London (UCL) has just started a preclinical study that will make it possible in the future to apply gene therapy to patients in a clinical trial. This project is done in collaboration with the Columbus…
TORONTO — A new gene therapy for an extremely rare disorder has offered two Canadian children a new chance at life, allowing them to move on their own after years of being trapped within their own bodies.The disease is called AAV deficiency, and known more commonly as ‘pediatric Parkinson’s’. It’s a debilitating genetic disorder that…